Rare Heroes Applauded at Annual Rare Disease Day Gala Celebration

- Despite advancements, improved access to orphan drugs still needed -

TORONTO, Feb. 28, 2013 /CNW/ - Last night at the Canadian Organization for Rare Disorders (CORD) Rare Disease Day Awards Gala Celebration, a dozen "rare heroes" were honoured for their contributions to the rare disease community in Canada.  Held on the eve of International Rare Disease Day, the event brought together the Canadian rare disease community as part of CORD's two-day Rare Disease Day Conference, with the hopeful theme of "From Worst to First: Transforming the Rare Disease Experience in Canada."

"Given the progress our community has made in the past year, we felt it was the right time to recognize those individuals and groups that have inspired, challenged, and forever changed what it means to live with a rare disorder," said Durhane Wong-Rieger, president of CORD.

Heroes of rare disease praised for contributions
In January 2013, CORD asked the rare disease community to nominate heroes in six Rarity Award categories, and then asked the community to vote on the nominees.  The winners of the CORD's first-ever Rarity Awards and Rare Honour Awards were announced at the Gala:

• Members of Parliament Kirsty Duncan and Joy Smith were recognized for their strong political leadership.
• David K. Lee from Health Canada received a standing ovation for his "dogged determination and tireless commitment" to drafting the orphan drug regulations and for ensuring they met "world class" standards.
• In the category of Patient Leadership, the community recognized CORD for its role as a "catalyst" in rare disease policy and creating a strong community.
• Dr. Cheryl Greenberg, geneticist at the Manitoba Institute of Child Health, received the top award for Health Professional Leadership.
• Dr. Lorne Greene from B.C. Children's Hospital was recognized for his contribution to Scientific Excellence.
• The Rarity Award of Honour went to the Rare Disease Foundation, led by Isabel Jordan.

CORD also surprised its president, Durhane Wong-Rieger, with an Outstanding Achievement Award for Volunteerism, recognizing six years of service. Five other individuals were recognized for their overall contributions to rare diseases. These included Barry Katsof, on behalf of the Canadian Association of PNH Patients, 12-year-old Jonathan Pitre, a hero and inspiration nominated by DEBRA Canada, Dr. Johanna Rommens, researcher at the Toronto Hospital for Sick Children, Maureen Sauve, on behalf of the Scleroderma Society of Canada, and Dr. Robert Schellenberg, who was nominated along with the Primary Immunodeficiency Clinic team at St. Paul's Hospital in Vancouver.

Awards were presented to the winners by CORD Board members including John Adams (Chair), Caroline Laughlin (Vice-Chair), Maureen Smith (Secretary), Charlene Doucette and Jim Mundy.  Additional Board members include Stephen McElroy (Treasurer), Kelly Gorman and Ashraf Ghadban.

"These awards demonstrate that rare disease heroes can be anyone. It can be a 12-year-old boy who, despite living with an incredibly painful disease, works tirelessly to raise awareness and funds for all those affected by it," said Wong-Rieger.  "It can be one of Canada's leading geneticists who, on Christmas Day, drove through a minus-30-degree snowstorm to pick up a mom and her 10-year-old son from Lebanon arriving in Winnipeg to start clinical trials for a bone disease that had already left the boy unable to walk.  These are our incredible everyday heroes!"

Improved access to orphan treatments urged
Despite major advancements made in the rare disease community, improved funding and access to treatments are still sought by CORD and rare disease advocates across the country.

In October 2012, Federal Health Minister Leona Aglukkaq announced the first Orphan Drug Regulatory Framework to improve access to new medicines for rare and neglected diseases.  This announcement came after years of advocacy by the rare disease community, nearly 30 years after the USA had passed the 1983 Orphan Drug Act, and 12 years after the EU passed similar legislation.

As an indication of the significance of orphan drug legislation, there had been only 10 drugs approved for rare diseases in the decade prior.  Since the legislation, there have been more than 400 orphan drugs approved. However, less than half of the orphan drugs approved by Health Canada are available to Canadian patients. Provincial drug plans have been reluctant to fund drugs for small patient populations, discouraging developers from bringing new rare disease drugs to Canada - despite the inclusion of Canadian patients in clinical trials. Additionally, federal incentives for research for rare disease drugs continue to lag.

"We are all inspired to work toward the implementation of the Orphan Drug regulations and to develop a universal program to ensure drugs are available to all appropriate patients across Canada," says Wong-Rieger. "What we are ultimately aiming for is a national Rare Disease Institute that can bring together all components of rare disease research, care and treatment and all stakeholders in the rare diseases community."

About CORD

CORD is Canada's national network for organizations representing all those with rare disorders. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare disorders in Canada.

SOURCE: Canadian Organization for Rare Disorders (CORD)

Media Contacts: 

Durhane Wong-Rieger
CORD President
Tel: [phone]
Email: [email protected]